Vertex’s Kalydeco Approved for Cystic Fibrosis in Infants At Least Four Months Old

September 28, 2020

The FDA has approved Vertex Pharmaceuticals’ Kalydeco (ivacaftor) to treat cystic fibrosis in children ages four months to less than six months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the drug.

The approval was based on a phase 3 clinical trial of six children between the ages of four to six months in which the drug demonstrated a similar safety profile to that seen in older children and adults.

Kalydeco is currently approved in the U.S. and European Union to treat cystic fibrosis in patients ages six months and older.

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